transfusion-dependent beta-thalassemia
Conditions
Brief summary
Proportion of Subjects achieving transfusion independence defined as a weighted average Hb ≥ 9.0 gr/dL without any red blood cell transfusion for a continuous period of ≥ 12 months (TI12) at any time during the study after the drug product administration. The assessment of TI12 starts 60 days after last RBC transfusion for post-transplant support or BTHAL standard of care.
Detailed description
Overall survival at 12 and 24 months post GT., Evaluation of the safety of treatment, Evaluation of biological correlates of safety
Interventions
DRUGAutologous CD34+ cells transduced with lentiviral vector encoding the human beta-globin gene
DRUGBusulfan Koanaa 6 mg/ml Konzentrat zur Herstellung einer Infusionslösung
DRUGPlerixafor Accord 20 mg/ml solution for injection
Sponsors
Fondazione Telethon Ets, San Raffaele Hospital
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of Subjects achieving transfusion independence defined as a weighted average Hb ≥ 9.0 gr/dL without any red blood cell transfusion for a continuous period of ≥ 12 months (TI12) at any time during the study after the drug product administration. The assessment of TI12 starts 60 days after last RBC transfusion for post-transplant support or BTHAL standard of care. | — |
Secondary
| Measure | Time frame |
|---|---|
| Overall survival at 12 and 24 months post GT., Evaluation of the safety of treatment, Evaluation of biological correlates of safety | — |
Countries
Italy
Outcome results
None listed